Multiple SclerosisNCT04540861
Managed Access Program (MAP) for Patients Diagnosed With Secondary Progressive Multiple Sclerosis With Active Disease
A study testing Siponimod in people with Multiple Sclerosis, run by Novartis Pharmaceuticals.
Trial summary
The purpose of this MAP Cohort Treatment Plan is to allow access to siponimod for eligible patients diagnosed with Secondary Progressive Multiple Sclerosis with active disease. The patient's Treating Physician should follow the suggested treatment guidelines and comply with all local health authority regulations. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Multiple Sclerosis
- What is being tested?
- Siponimod
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients eligible for inclusion in this Treatment Plan have to meet all of the following criteria:
- Adult patients who are able to provide written informed consent and have done so Note: proof of efficacy in the Phase III EXPAND study (Kappos et al 2018) has been obtained in patients who were aged 18 to 61 years (inclusive) at screening
- Diagnosis of SPMS with active disease. The SPMS diagnosis must follow an initial relapsing-remitting disease course of multiple sclerosis (MS) and align with the diagnostic criteria published (Lublin and Reingold 1996, Rovaris et al 2006, Lublin et al 2014)
- Patient is ambulatory, i.e., has an EDSS score of ≤6.5
- Patient has no comparable or satisfactory alternative therapy available for treatment of SPMS Written patient informed consent must be obtained prior to start of treatment. If consent cannot be expressed in writing, it must be formally documented and witnessed, ideally via an independent trusted witness.
Who cannot join
- Patients eligible for this Treatment Plan must not meet any of the following criteria:
- Disease exclusions
- Relapsing-remitting multiple sclerosis
- Primary progressive multiple sclerosis
- Secondary progressive multiple sclerosis without active disease
- Medical conditions precluding inclusion such as lab abnormalities or underlying diseases
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- Patients with an active or stable but treated chronic disease of the immune system other than MS (e.g. rheumatoid arthritis, scleroderma, Sjogren's syndrome, Crohn's disease, ulcerative colitis, etc.) or with a known immunodeficiency syndrome (AIDS, hereditary immune deficiency, drug-induced immune deficiency)
- Severe active infections Patients affected by severe infections should only be included after full resolution of the condition
- Second-degree AV block Mobitz type II or higher, sick-sinus syndrome, sino-atrial heart block or significant QT prolongation (QTc ≥500 msec).
- History of cardiac arrest, recurrent syncope, symptomatic bradycardia, severe cerebrovascular disease, uncontrolled hypertension or severe untreated sleep apnea.
- Significant liver disease
- Ongoing macular edema (patients with a history of resolved macular edema are allowed to enter the program)
- Patients homozygous for CYP2C9*3 (CYP2C9*3*3 genotype)
- Patients without a health-care professional confirmed history of chickenpox or documentation of a full course of vaccination with varicella vaccine. They have to undergo antibody testing to varicella zoster virus (VZV) before initiating treatment with siponimod. A full course of vaccination for antibody-negative patients with varicella vaccine is required prior to commencing treatment with siponimod. Initiation of treatment with siponimod has be postponed for one month after completion of the vaccination course to allow full effect of vaccination to occur.
- History of hypersensitivity to any drugs or metabolites of similar chemical classes as siponimod.
- Prior therapy precluding enrollment:
- Natalizumab, immunosuppressive/chemotherapeutic medications (e.g. azathioprine, methotrexate) within 6 months prior to enrollment
- Rituximab, ofatumumab, ocrelizumab, cladribine, or cyclophosphamide within one year prior to enrollment
- Any mitoxantrone during previous two years prior to enrollment or evidence of cardiotoxicity following mitoxantrone or a cumulative life-time dose of more than 60 mg/m2
- alemtuzumab, lymphoid irradiation, bone marrow transplantation or other immunosuppressive treatments with effects potentially lasting over 6 months, at any time
- Concomitant therapy precluding enrollment:
- Alemtuzumab, azathioprine, cyclophosphamide, cyclosporine, methotrexate, mitoxantrone, natalizumab, rituximab, ofatumumab, or ocrelizumab as immunosuppressive or immune-modulating therapies
- Only during treatment initiation: Class Ia (e.g. quinidine, procainamide), Class III anti-arrhythmic drugs (e.g. amiodarone, sotalol) during treatment initiation
- Have any of the following out-of-range laboratory values:
- Alanine aminotransferase (ALT) or aspartate aminotransferase (AST), or gamma-glutamyl transferase (GGT) greater than 5 times the upper limit of normal (ULN) range
- White blood cell (WBC) count < 3,500/mm3 (< 3.5 x 109/L)
- Lymphocyte count < 800/mm3 (< 0.8 x 109/L)
- Serum potassium > ULN
- Participation in a prior investigational study within 30 days prior to enrollment or within five half-lives of the investigational study drug or until the expected pharmacodynamic effect has returned to baseline, whichever is longer
- Pregnancy statements and contraception requirements
- Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test (> 5 mIU/mL).
- Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, UNLESS they are:
- Women whose partners have been sterilized by vasectomy or other means.
- Using a highly effective method of birth control for the duration of treatment with siponimod and for at least ten days after stopping treatment with siponimod. Highly effective method of birth control are such resulting in a less than 1% per year failure rate when used consistently and correctly, such as implants, injectables, combined oral contraceptives, and some intrauterine devices (IUDs); periodic abstinence (e.g. calendar, ovulation, symptothermal, post-ovulation methods) is not acceptable.
- Total abstinence from female-male intercourse (when this is in line with the preferred and usual lifestyle of the subject, if accepted by local regulation)
- Not able to understand and to comply with treatment instructions and requirements.
About this study
Official title: Managed Access Program (MAP) to Provide Access to Siponimod Treatment for Patients Diagnosed With Secondary Progressive Multiple Sclerosis With no Satisfactory Alternative Treatment
The purpose of this MAP Cohort Treatment Plan is to allow access to siponimod for eligible patients diagnosed with Secondary Progressive Multiple Sclerosis with active disease.
The patient's Treating Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.
The requesting Treating Physician should submit a request for access to the drug (often referred to as Compassionate Use) to Novartis which will be reviewed and assessed by the medical team experienced with the drug and indication.
- Conditions
- Multiple Sclerosis
- Being tested
- Siponimod
- Keywords
- SPMS with active disease, SPMS, Secondary Progressive Multiple Sclerosis, siponimod, Mayzent
- Medical subject headings
- Multiple Sclerosis, Multiple Sclerosis, Chronic Progressive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SiponimodDrug
Also called Mayzent
1 or 2 mg film-coated tablets. The tablets have to be taken orally once daily and swallowed whole with water, with or without food.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Sep 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Multiple SclerosisStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04540861
- Sponsor's protocol ID
- CBAF312A2001M
- Status last verified
- Nov 2025
- First submitted
- 31 Aug 2020
- Last updated
- 4 Dec 2025
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Managed Access Program (MAP) to Provide Access to Siponimod Treatment for Patients Diagnosed With Secondary Progressive Multiple Sclerosis With no Satisfactory Alternative Treatment
- ClinicalTrials.gov ID
- NCT04540861
- Acronym / sponsor ID
- CBAF312A2001M
- Conditions
- Multiple Sclerosis
- Keywords
- SPMS with active disease, SPMS, Secondary Progressive Multiple Sclerosis, siponimod, Mayzent
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- First posted
- 7 Sep 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this MAP Cohort Treatment Plan is to allow access to siponimod for eligible patients diagnosed with Secondary Progressive Multiple Sclerosis with active disease.
The patient's Treating Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.
The requesting Treating Physician should submit a request for access to the drug (often referred to as Compassionate Use) to Novartis which will be reviewed and assessed by the medical team experienced with the drug and indication.
Study design
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Patients eligible for inclusion in this Treatment Plan have to meet all of the following criteria:
- Adult patients who are able to provide written informed consent and have done so Note: proof of efficacy in the Phase III EXPAND study (Kappos et al 2018) has been obtained in patients who were aged 18 to 61 years (inclusive) at screening
- Diagnosis of SPMS with active disease. The SPMS diagnosis must follow an initial relapsing-remitting disease course of multiple sclerosis (MS) and align with the diagnostic criteria published (Lublin and Reingold 1996, Rovaris et al 2006, Lublin et al 2014)
- Patient is ambulatory, i.e., has an EDSS score of ≤6.5
- Patient has no comparable or satisfactory alternative therapy available for treatment of SPMS Written patient informed consent must be obtained prior to start of treatment. If consent cannot be expressed in writing, it must be formally documented and witnessed, ideally via an independent trusted witness.
Key exclusion criteria (36 listed)
- Disease exclusions
- Medical conditions precluding inclusion such as lab abnormalities or underlying diseases
- Prior therapy precluding enrollment:
- Concomitant therapy precluding enrollment:
- Have any of the following out-of-range laboratory values:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SiponimodAlso: Mayzent1 or 2 mg film-coated tablets. The tablets have to be taken orally once daily and swallowed whole with water, with or without food. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Dec 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
Questions about this trial
What is this clinical trial studying?
A study testing Siponimod in people with Multiple Sclerosis, run by Novartis Pharmaceuticals.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 36 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 4 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Dec 2025.